Regulatory
Rapporteur
Write to the Editor at publications@topra.org
July/August 2025 | Vol. 22 | No. 7
EDITORIAL
Patient voice: Shaping the future of medicine regulation
The July/August issue of Regulatory Rapporteur focuses on patient involvement.
Patients are at the heart of everything regulatory professionals do. Giving patients a voice and opportunities for collaboration can improve health outcomes, as well as providing a better treatment experience. In this issue, we look at the growing levels of patient involvement in regulatory processes and how these can influence new medicine applications, patient information and guidance generation.
By Victoria Goff
FOCUS
The evolving landscape of public and patient involvement
Public and patient involvement is known under many different terms and this lack of a harmonised definition can create confusion over its aims. This article gives an overview of the role of the public and patients across research, academia, healthcare and industry, through a lens of personal experience.
By Mandy Daly of the Irish Neonatal Health Alliance
FOCUS
Patient involvement in the regulatory area: Insights into national level dialogues
Patients offer unique insights into the challenges of living with conditions, which provide regulators with better awareness when making decisions about medicinal product approvals. While the European Medicines Agency’s patient involvement processes are now well documented, less is known about the practices of national competent authorities (NCAs) across different EU Member States.
This article provides an overview of the findings of a survey undertaken by the European Patients’ Academy on Therapeutic Innovation (EUPATI) to better understand the different levels of patient involvement among European NCA regulatory processes and makes recommendations on how this can be improved.
By Maria Dutarte of EUPATI and Birka Lehman, Independent expert, formerly of the Federal Institute for Drugs and Medical Devices (BfArM)
FOCUS
The importance of patient advocacy in the EMA ODD process
This article examines the critical role of patient advocacy groups in the orphan drug designation (ODD) pathway and the challenge of ensuring patient voices are heard by the European Medicines Agency (EMA). It highlights practical sponsor strategies for leveraging the EMA engagement framework to define unmet needs, influence regulatory decisions and drive more patient-centred outcomes.
By Catherine Moncad of TMC Pharma Services Limited
FOCUS
Realising opportunities to generate RWE for regulatory decisions through randomised pragmatic trials
This article summarises a European Federation of Pharmaceutical Industries and Associations (EFPIA) paper on the potential opportunities of randomised pragmatic trials and how they bridge traditional randomised clinical trials and non-interventional studies. It explains how this type of study can be a valuable tool for evidence generation and can contribute to more patient-centric drug development.
By EFPIA
STANDALONE
Diversity in clinical research: Regulatory considerations and updates
This article discusses the importance of heterogeneity in clinical research and the impact this has on regulatory compliance, market access and the understanding of benefit-to-risk ratios. It offers an overview of the regulations and guidance in this area and offers strategic approaches that can be used by sponsors to promote diversity in clinical trials.
By Heidi Stauffer-McDonald and Sean Schofield of the PPD™ clinical research business of Thermo Fisher Scientific
STANDALONE
FDA comparability guidance: The impact of cell and gene therapy products
This article reviews the US Food and Drug Administration (FDA) draft guidance for manufacturing changes and comparability of cellular and gene therapy products. It compares the proposed changes in this new guidance against existing guidance available for this product type and identifies key changes that will need to be considered by manufacturers and regulatory teams moving forward.
By Laura Guijarro, Rajendra Kunda, Slobodanka Cirin-Varadjan and Hao Liu of Syneos Health
TOPRA is introducing a new Patient Engagement Special Interest Network (SPIN) group. Please contact gloria.dwyer@topra.org to get involved.
Topics
- Cell and gene therapy (CGT)
- Clinical Trials
- Diversity
- European Medicines Agency (EMA)
- European Patients' Academy on Therapeutic Innovation (EUPATI)
- Orphan drug designation (ODD)
- patient advocacy
- Patient involvement
- Patient voice
- Pharmaceuticals
- Public and Patient Involvement
- randomised pragmatic trials
- Real world evidence (RWE)
- US Food and Drug Administration (FDA)
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